Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global
commercial-stage biopharmaceutical company focused on transforming
the lives of patients and their families living with rare
neuroendocrine diseases, today provided a business update and
announced it has completed screening for enrollment in the pivotal,
Phase 3 clinical trial evaluating setmelanotide in patients with
acquired hypothalamic obesity.
Rhythm closed screening and anticipates overenrolling the
ongoing phase 3 hypothalamic obesity trial with more than 140
patients consented and going through active screening and baseline
assessments. In this trial, patients with acquired hypothalamic
obesity aged 4 years or older will be randomized 2:1 to
setmelanotide therapy or placebo for a total of 60 weeks, including
up to eight weeks for dose titration. The Company expects to obtain
top-line study results in H1 2025.
“2023 was a truly transformational year for Rhythm, as we made
excellent progress with the execution of our IMCIVREE®
(setmelanotide) commercial plan while also advancing important
development efforts to expand the number of patients living with
hyperphagia and severe obesity that could potentially benefit from
this therapy,” said David Meeker, M.D., Chair, President and Chief
Executive Officer of Rhythm. “We believe the rapid over enrollment
in less than one year and execution of our Phase 3 hypothalamic
obesity trial is a strong indicator of the unmet need for an
effective therapy for patients who have no approved therapeutic
options and the community’s excitement around setmelanotide’s
potential to make a meaningful difference in their lives.”
Positive Reimbursement Decisions Achieved in
SpainRhythm today also announced that federal healthcare
authorities in Spain have approved reimbursement for IMCIVREE®
(setmelanotide) for the treatment of obesity and control of hunger
associated with Bardet-Biedl syndrome (BBS) or biallelic
proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin
type 1 (PCSK1), or biallelic leptin receptor (LEPR) deficiency.
“We are pleased that the Ministry of Health in Spain has
recognized the treatment needs of patients with hyperphagia and
early-onset obesity of BBS and POMC/LEPR deficiencies and IMCIVREE
as the first and only precision medicine for patients with these
diseases,” said Yann Mazabraud, Executive Vice President and Head
of International at Rhythm. “We are excited to achieve access in
this important market and deliver IMCIVREE to patients and
families.”
BBS and POMC, PCSK1 and LEPR deficiencies are rare diseases with
hallmark symptoms of hyperphagia, a pathological hunger that leads
to abnormal food-seeking behaviors, and early-onset, severe
obesity. BBS also is associated with cognitive impairment,
polydactyly, renal dysfunction, hypogonadism, and visual
impairment. POMC or LEPR deficiency are ultra-rare diseases with
very few patients identified. With multiple symptoms as part of a
syndrome, BBS patients, while still under-diagnosed, are more
easily identified. Rhythm estimates the prevalence of BBS to be
approximately 700 people in Spain with approximately 100 patients
identified.
RM-718 IND Accepted by the FDARhythm also
announced today that the FDA accepted the Company's Investigational
New Drug (IND) application for RM-718, a new, weekly treatment
designed to be more targeted and potent than setmelanotide with the
potential to not cause hyperpigmentation. The Company anticipates
beginning Phase 1 in-human trials in the first half of 2024,
including a multiple-ascending dose study in patients with
hypothalamic obesity.
About Rhythm Pharmaceuticals Rhythm is a
commercial-stage biopharmaceutical company committed to
transforming the lives of patients and their families living with
rare neuroendocrine diseases. Rhythm’s lead asset, IMCIVREE®
(setmelanotide), an MC4R agonist designed to treat hyperphagia and
severe obesity, is approved by the FDA for chronic weight
management in adult and pediatric patients 6 years of age and older
with monogenic or syndromic obesity due to pro-opiomelanocortin
(POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or
leptin receptor (LEPR) deficiency confirmed by genetic testing, or
patients with a clinical diagnosis of Bardet-Biedl syndrome (BBS).
Both the European Commission (EC) and the UK’s Medicines &
Healthcare Products Regulatory Agency (MHRA) have authorized
setmelanotide for the treatment of obesity and the control of
hunger associated with genetically confirmed BBS or genetically
confirmed loss-of-function biallelic POMC, including PCSK1,
deficiency or biallelic LEPR deficiency in adults and children 6
years of age and above. Additionally, Rhythm is advancing a
broad clinical development program for setmelanotide in other rare
diseases, as well as a preclinical suite of small molecules for the
treatment of congenital hyperinsulinism. Rhythm’s headquarters is
in Boston, MA.
Setmelanotide Indication In the United
States, setmelanotide is indicated for chronic weight management in
adult and pediatric patients 6 years of age and older with
monogenic or syndromic obesity due to POMC, PCSK1 or LEPR
deficiency as determined by an FDA-approved test demonstrating
variants in POMC, PCSK1 or LEPR genes that are interpreted as
pathogenic, likely pathogenic, or of uncertain significance (VUS)
or BBS.
In the European Union, setmelanotide is indicated for the
treatment of obesity and the control of hunger associated with
genetically confirmed Bardet-Biedl syndrome (BBS) or genetically
confirmed loss-of-function biallelic proopiomelanocortin (POMC),
including PCSK1, deficiency or biallelic leptin receptor (LEPR)
deficiency in adults and children 6 years of age and
above.
Limitations of Use In the United
States and Europe, Setmelanotide should be prescribed and
supervised by a physician with expertise in obesity with underlying
genetic etiology.
Setmelanotide is not indicated for the treatment of patients
with the following conditions as setmelanotide would not be
expected to be effective:
- Obesity due to suspected POMC, PCSK1 or LEPR deficiency
with POMC, PCSK1 or LEPR variants classified as benign or likely
benign
- Other types of obesity not related to POMC, PCSK1 or LEPR
deficiency, or BBS, including obesity associated with other genetic
syndromes and general (polygenic) obesity.
WARNINGS AND PRECAUTIONS
Skin Monitoring: Setmelanotide may lead to
generalized increased skin pigmentation and darkening of
pre-existing naevi because of its pharmacologic effect. Full body
skin examinations should be conducted annually to monitor
pre-existing and new skin pigmentary lesions before and during
treatment with setmelanotide.
Heart rate and blood pressure monitoring: Heart
rate and blood pressure should be monitored as part of standard
clinical practice at each medical visit (at least every 6 months)
for patients treated with setmelanotide.
Prolonged penile erection: Spontaneous penile
erections have been reported in clinical trials with setmelanotide.
Patients who have a penile erection lasting longer than 4 hours
should be instructed to seek emergency medical attention for
potential treatment of priapism.
Depression: In clinical trials, depression has
been reported in patients treated with setmelanotide. Patients with
depression should be monitored at each medical visit during
treatment with setmelanotide. Consideration should be given to
discontinuing setmelanotide if patients experience suicidal
thoughts or behaviors.
Pediatric Population: The prescribing physician
should periodically assess response to setmelanotide therapy. In
growing children, the impact of weight loss on growth and
maturation should be evaluated. The prescribing physician should
monitor growth (height and weight) using age- and sex-appropriate
growth curves.
Excipients: This medicinal product contains 10
mg benzyl alcohol in each ml. Benzyl alcohol may cause allergic
reactions. Patients who are pregnant or breastfeeding should be
advised of the potential risk from the excipient benzyl alcohol,
which might accumulate over time and cause metabolic acidosis. This
medicinal product should be used with caution in patients with
hepatic or renal impairment, because of the potential risk from the
excipient benzyl alcohol which might accumulate over time and cause
metabolic acidosis.
Sodium: This medicinal product contains less
than 1 mmol sodium (23 mg) per dose, that is to say essentially
“sodium-free.”
ADVERSE REACTIONS The most frequent
adverse reactions are hyperpigmentation (51%), injection site
reaction (39%), nausea (33%), and headache (26%).
USE IN SPECIFIC POPULATIONS
Pregnancy There are no data from the use
of setmelanotide in pregnant women. Animal studies do not indicate
direct harmful effects with respect to reproductive toxicity.
However, administration of setmelanotide to pregnant rabbits
resulted in decreased maternal food consumption leading to
embryo-fetal effects. As a precautionary measure, setmelanotide
should not be started during pregnancy or while attempting to get
pregnant as weight loss during pregnancy may result in fetal harm.
If a patient who is taking setmelanotide has reached a stable
weight and becomes pregnant, consideration should be given to
maintaining setmelanotide treatment as there was no proof of
teratogenicity in the nonclinical data. If a patient who is taking
setmelanotide and still losing weight gets pregnant, setmelanotide
should either be discontinued, or the dose reduced while monitoring
for the recommended weight gain during pregnancy. The treating
physician should carefully monitor weight during pregnancy in a
patient taking setmelanotide.
Breast-feeding It is unknown whether
setmelanotide is excreted in human milk. A nonclinical study showed
that setmelanotide is excreted in the milk of nursing rats. No
quantifiable setmelanotide concentrations were detected in plasma
from nursing pups. A risk to the newborn/infant cannot be excluded.
A decision must be made whether to discontinue breastfeeding or to
discontinue/abstain from setmelanotide therapy taking into account
the benefit of breastfeeding for the child and the benefit of
therapy for the mother.
Fertility No human data on the effect of
setmelanotide on fertility are available. Animal studies did not
indicate harmful effects with respect to fertility.
To report SUSPECTED ADVERSE REACTIONS, contact Rhythm
Pharmaceuticals at +1 (833) 789-6337. See Summary of Product
Characteristics’ APPENDIX V for a list of European national
reporting systems to communicate adverse reactions.
Please see the full Prescribing Information for
additional Important Safety Information.
Forward-Looking Statements This press
release contains forward-looking statements within the meaning of
the Private Securities Litigation Reform Act of 1995. All
statements contained in this press release that do not relate to
matters of historical fact should be considered forward-looking
statements, including without limitation statements regarding the
potential, safety, efficacy, and regulatory and clinical progress
of setmelanotide, including the anticipated timing for initiation
of clinical trials and release of clinical trial data and our
expectations surrounding potential regulatory submissions,
approvals and timing thereof, including for RM-718 and
setmelanotide, our business strategy and plans, including regarding
commercialization of setmelanotide in the United States and Europe,
the application of genetic testing and related growth potential,
and expectations surrounding the potential market opportunity for
our product candidates. Statements using word such as “expect”,
“anticipate”, “believe”, “may”, “will”, "aim" and similar terms are
also forward-looking statements. Such statements are subject to
numerous risks and uncertainties, including, but not limited to,
risks relating to our liquidity and expenses, our ability to enroll
patients in clinical trials, the design and outcome of clinical
trials, the ability to achieve necessary regulatory approvals,
risks associated with data analysis and reporting, failure to
identify and develop additional product candidates, unfavorable
pricing regulations, third-party reimbursement practices or
healthcare reform initiatives, risks associated with the laws and
regulations governing our international operations and the costs of
any related compliance programs, the impact of competition, risks
relating to product liability lawsuits, inability to maintain our
collaborations, or the failure of these collaborations, our
reliance on third parties, risks relating to intellectual property,
our ability to hire and retain necessary personnel, the impact of
the COVID-19 pandemic and general economic conditions on our
business and operations, including our preclinical studies,
clinical trials and commercialization prospects, failure to realize
the anticipated benefits of our acquisition of Xinvento B.V. or
significant integration difficulties related to the acquisition,
and the other important factors discussed under the caption “Risk
Factors” in our Quarterly Report on Form 10-Q for the quarter ended
September 30, 2023 and our other filings with the Securities and
Exchange Commission. Except as required by law, we undertake no
obligations to make any revisions to the forward-looking statements
contained in this release or to update them to reflect events or
circumstances occurring after the date of this release, whether as
a result of new information, future developments or
otherwise.
Corporate Contact:David ConnollyExecutive
Director, Investor Relations and Corporate CommunicationsRhythm
Pharmaceuticals, Inc.857-264-4280dconnolly@rhythmtx.com
Media Contact:Adam DaleyBerry & Company
Public Relations212-253-8881adaley@berrypr.com
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